Genetics

The genome is the one component uniting the countless number of cells in the body. No matter how disparate their function or appearance, every cell is controlled by the genome. However, there are downsides to this level of control. A person may be born with a genetic disorder, such as cystic fibrosis or Huntington’s disease which cuts their life short or causes severe disability. Even if a person is born with a perfectly healthy genome, the copies of their genome in each cell may be mutated over time. These mutations can lead to illnesses, the most prominent of which is cancer.

Therefore, The Cure Is Now is dedicated to the study of genetics, in order to determine the ways in which genes control cells (with an eye towards finding potential targets for intervention in case of disease) and methods to protect the genome from damage and to repair any pathologies, whether they are inherited or arose spontaneously.

Immuno-Therapy

The immune system is one of the most sophisticated and powerful parts of the human body. When a new pathogen enters the bloodstream, immune cells quickly develop antibodies that bind to the pathogen more tightly than any molecule that humans are capable of engineering, and they do so more quickly than any lab could design such a molecule. Such a powerful system would have obvious applications towards fighting noninfectious diseases, such as cancer. On the other hand, dysregulation of the immune system is responsible for autoimmune disorders such as type I diabetes. Therefore, exploring ways in which the immune system can be harnessed, stimulated or, if necessary, suppressed is an important area of research.

Over time, some diseases, such as cancer, Alzheimer’s and rheumatoid arthritis, increase in prevalence. Curing these diseases would enable senior citizens to pursue a happy, healthy and independent lifestyle.

Aubrey de Grey is one of the leading visionaries in research on age-related diseases. His achievements include the enumeration of the types of damage that cause age-related diseases and the development of the Strategies for Engineered Negligible Senescence (SENS), which address each type of damage. The types of age-related damage identified by Aubrey de Grey are:

Cell loss and atrophy: repairing this damage would lead to a cure for osteoporosis and the loss of strength in the elderly.

Cell mutations and epimutations: repairing this damage would lead to a cure for cancer.

Mitochondrial mutations: repairing this damage would cure age-related degenerative disease, as well as hereditary diseases, such as Leber’s hereditary optical neuropathy.

Accumulation of intracellular junk: repairing this damage would lead to cures for macular degeneration and Alzheimer’s disease.

Accumulation of extracellular junk: repairing this damage would also contribute to a cure for Alzheimer’s; it would also help reduce the prevalence of cardiovascular disease.

Extracellular crosslinks: repairing this damage would help treat cataracts and improve mobility in the elderly.

Most therapies targeted towards these diseases merely treat the symptoms, while leaving the damage that is the underlying cause untouched. Ultimately, such therapies are severely limited in their ability to permanently and profoundly treat disease. The Cure is Now aims to repair the damage at the root of these diseases to produce true cures. Click here to lean more about Dr. de Grey’s research projects.

Silvia Gravina studies epigenomic mutations. The epigenome consists of modifications to the nucleotides that make up the genome. The pattern of these modifications influences which genes are expressed, at what times and at what levels. If they become less organized with age, then the intricate pattern of gene expression that ensures health may be disrupted. Studying how the epigenome changes with age may help to treat diseases in elderly individuals.

Bill Andrews studies the regulation of telomere length. Telomeres are stretches of DNA sequences at the end of each chromosome that protect it from damage. With each cell division, however, the telomeres get shorter. If they get too short, the cells may stop dividing; if they do keep dividing, then the rest of the genome, no longer protected by telomeres, is vulnerable to damage. Either way, the shortening of telomeres appears to be associated with a decline in health in the elderly. There is an enzyme, called telomerase, which lengthens telomeres in humans, but its activity is normally too low to prevent telomere shortening. Currently, Bill Andrews focuses on finding drugs to increase the activity of telomerase and restore telomere length. The Cure is Now is interested in supporting Bill Andrews in his research due to its applications to treating multiple diseases. Click here to lean more about Dr. Andrews’ research projects.

Brandon Milholland studies the role of DNA damage and mutation in the aging process. At birth, every cell in the body has an identical copy of the genome. Every time a cell divides, however, there is an opportunity for errors to be introduced. Over decades and many cell divisions, these errors can accumulate. The result is that in old individuals, every cell may have a different copy of the genome, each with its own set of errors. These mutations can cause cells to decline in function, but the most pressing consequence is the possibility that a cell may receive a mutation that causes it to become cancerous. Studying how cells become mutated may help in developing ways to treat or even prevent cancer and other age-related diseases. Brandon Milholland serves as The Cure is Now’s Research Coordinator to identify promising fields of research and possible collaborations between other scientists working for The Cure is Now. Click here to lean more about Brandon’s research projects.

Gene Therapy

In the past, treatments for diseases have consisted of manipulating the cellular and physiological environment, while leaving the genome unchanged. While this approach has proved effective for many diseases, especially those that result from external factors, such as infection, it falls short when applied to diseases whose root cause lies in the genome. The result is that many people, born with genetic diseases through no fault of their own, have had few or no treatments available to them. Recent advances have made it possible to manipulate the genome of human cells, and there is now hope that debilitating genetic diseases will be cured in the near future.

Systems Biology

The components of biological systems do not exist in isolation. Rather, they are constantly interacting with each other. Previously, biology was studied in a reductionist manner, one piece at a time. As our understanding has increased, however, it has become apparent that further advances require an integrationist approach, in which systems as a whole are studied. It is only by studying the complex networks of interaction that we can begin to understand how organisms truly work, and how to cure complex diseases.

Tanya Petrossian uses computational and biochemical techniques to study the methyltransferome, the system by a chemical signal, the methyl group, is transferred between different molecules in the cell.